Trends & Insights, January 2026: A pharmaphorum industry report

Outside of what we've been reporting on in terms of news and in-depth features, interviews, and in-person event coverage, behind the headlines the year also opened with a flurry of regulatory milestones, ophthalmology advances, AI-first R&D announcements, and tangible investments in manufacturing and packaging capacity.

Amid this, oncology continued to dominate outcomes news, but two cross cutting currents stood out: (1) platform plays – in gene editing, reprogramming, and AI-guided biology – moving from promise to clinic, and (2) operations realism – companies spending on fill finish, blister innovations, and digital tools to lower cost and boost resilience.

Below, we’ve brought together the month’s themes in our new Trends Outside the Headlines, trends & insights report, collecting together and assessing the signals that matter for life sciences operators, and what to watch in Q1–Q2.

1. Translating platform science into the clinic

Cellular rejuvenation enters human testing. Life Biosciences secured FDA IND clearance for ER 100, the first clinical-stage therapy using partial epigenetic reprogramming (OSK factors) to treat optic neuropathies (OAG and NAION). The first in human Phase 1 study (NCT07290244) starts in Q1, reflecting years of intravitreal preclinical work and controlled OSK expression readouts in primates. This is a defining moment for longevity modalities, and noteworthy for starting in ophthalmology – clear delivery route, functional end points, and manageable risk.

Gene therapy broadens its sights in the eye. Complement Therapeutics gained FDA Fast Track for CTx001 in geographic atrophy (GA); the AAV-based construct delivers a mini CR1 to modulate classical and alternative complement pathways, with first dosing targeted for Q1 in the Opti GAIN Phase I/II trial. Meanwhile, AAVantgarde flagged a JPM presentation update spanning Stargardt’s and Usher 1B programmes, and Axol Bioscience raised $2.8M to scale iPSC disease models – feeding discovery and translational work across neuro ophthalmic indications. Expect 2026 to be an execution year for gene therapy in retinal disease, with complementary modalities (gene, reprogramming, cell models) converging on clearer efficacy signals.

AI-designed biology is now therapeutic-facing. Basecamp Research unveiled EDEN evolutionary models and an aiPGI platform that designed programmable gene insertion enzymes for large DNA integration at precise sites, plus a 97% wet lab success rate designing antimicrobial peptides – positioning AI not just as an enhancer, but as a generator of novel therapeutic effectors. If reproducibility holds, this creates a new lane alongside CRISPR nucleases and integrases.

Precision psychiatry inches from markers to medicines. HMNC Brain Health highlighted OLIVE trial findings: a genetically defined subgroup (~27%) with superior response to investigational antidepressant BH 200. The press focus – validating biomarkers that translate to clinical utility – speaks to payer needs and labelable subgroups, rather than exploratory omics alone.

2. Ophthalmology: A hotbed of first in class moves

Partial reprogramming (ER 100) into Phase 1 for glaucoma/NAION marks the first clinical test of controlled OSK in humans. 

GA fast tracked: Complement Therapeutics’ CTx001 secured Fast Track and IND clearance; first in human Opti GAIN study is imminent. 

Market expansion in med derm cross over: Almirall’s Seysara (sarecycline) won NMPA approval for moderate to severe acne (≥9 years), with China commercial launch targeted for 2026 – relevant to dermatology–ophthalmology intersections (antibiotic stewardship, ocular tolerability of systemic agents). 

Device/packaging underpinning adherence: Nemera’s preservative free Novelia eyedropper and ACG’s SuperPod blister (smaller cavities, full barrier) highlight how design can cut carbon, improve usability, and protect moisture sensitive ophthalmics. 

3. Oncology remains the proof engine for value

• Checkpoint plus chemo in a rare cancer: Incyte received a positive CHMP opinion for Zynyz (retifanlimab) + carboplatin/paclitaxel as first line therapy in advanced SCAC – potentially the first PD 1 regimen in Europe for this population. Phase 3 (POD1UM 303/InterAACT2) showed a 37% reduction in risk of progression/death and median PFS 9.3 vs 7.4 months; EC decision pending.
• Multiple myeloma deep responses continue: Press activity summarised robust MRD negativity and CR rates with D VRd (DARZALEX FASPRO) in NDMM (CEPHEUS). While label nuances differ by region, depth and durability benchmarks keep rising, reinforcing CD38 backbone strategies.
• Emerging ADCs and degraders: Tubulis’ TUB 040/030 and Ascentage’s APG 3288 (BTK degrader) show steady modality diversification beyond antibodies and TKIs – important for tumours with resistance pathways.

4. Imaging and diagnostics: Lower dose, higher fidelity, and earlier minimal disease detection

Low dose MRI contrast: Bayer’s gadoquatrane Phase III program (QUANTI) advances filings across regions with a 60% reduction in gadolinium dose vs macrocyclic GBCAs at 0.1 mmol/kg, with new subgroup data (abdominal, cardiac, MRA, CNS). If approved, this is a rare step change in GBCA dosing strategy with workflow and safety implications. 

ctDNA MRD mainstreaming: SAGA Diagnostics’ Pathlight MRD and other liquid biopsy solutions signal a move toward routine, tumour agnostic surveillance and trial enrichment. Coupled with AI enabled trial design, sponsors are tightening Phase 2–3 timelines.

5. AI in life sciences: From pilots to platform commitments

• Designing medicines, not just models: Basecamp’s EDEN and aiPGI exemplify AI as a creator of therapeutic enzymes and antimicrobial peptides with high lab validation rates – an inflection beyond in silico screening.
• Precision analytics at scale: SOPHiA GENETICS passed 2 million genomic profiles analyzed on DDM and forged an MD Anderson collaboration – evidence that AI genomics networks are achieving clinical grade scale.
• Operational AI adoption pressure: Survey data (Argon & Co) showed 49% of life sciences leaders plan to invest in AI for efficiencies amid rising input costs and supply risk – pairing nicely with digital twins and predictive analytics in manufacturing/logistics.

6. Manufacturing, packaging, and supply resilience: Quiet investments with big payoffs

• Blister innovation with sustainability math: ACG’s SuperPod reduces cold form blister cavity size by up to 39% while keeping full barrier performance and line runnability – translating into smaller packs, lower materials/CO₂, and higher “blisters per shot”. For moisture sensitive and oncology products, reduced headspace also improves protection.
• Sterile fill finish capacity building: Multiple announcements (e.g., Sharp Sterile, Symbiosis, Vetter pipeline investments) underscore continued demand for high mix, small batch biologics and gene therapy fill finish, including tamper evident syringe systems.
• iPSC manufacturing platforms: Cellistic and Axol moves point to industrialisation of iPSC models and, over time, allogeneic cell therapy inputs, with knock on effects for QC analytics and release testing.
• Orthopedics infection management: Biocomposites rolled out SYNICEM preformed antibiotic loaded spacers in Canada (after a US launch), reflecting continued demand for ready to use, standardised devices that save OR time and reduce mechanical risk in two stage revisions.

7. Market access and global expansion: Dermatology, rare oncology, and China momentum

• China dermatology market opening: Almirall’s Seysara (sarecycline) approval by NMPA (ages ≥9) sets up 2026 launch via Sinomune, highlighting both the demand for narrow spectrum acne agents and the benefits of local partnerships for distribution scale.
• Rare oncology precedent: If the European Commission affirms the CHMP opinion on Zynyz for SCAC, it establishes a PD 1–based first line standard in Europe and a reference point for other rare GI oncology filings.

8. Neuroscience: Mechanism clues and clinical pragmatism

• Diet–receptor–seizure link: New academic findings connected the ketogenic diet’s β hydroxybutyrate to HCAR2 receptor activity in hippocampal circuits and microglia, suggesting druggable mechanisms (e.g., niacin) that could mimic keto benefits for refractory epilepsy – illustrative of translational paths industry can pick up.
• iPSC disease models scale out: Axol’s funding supports expanded ALS/Huntington’s platforms – feeding both target discovery and functional screening as wet lab complements to AI predictions.

9. Anti microbial resistance (AMR) and vaccines: Cautious optimism

• AI generated AMPs: Basecamp’s 97% lab active antimicrobial peptides, including hits against multidrug resistant pathogens, offer a rare shot of optimism in AMR discovery.
• Broad pneumococcal concepts: ReNewVax reported universal pneumococcal vaccine progress (100% efficacy in mice), while TB programme TBD09 advanced with expedited designations, signalling that non COVID infectious disease R&D is regaining investor and regulatory attention.

10. Capital flows: Selective, but supportive

January funding skewed toward tools/enablers and clear clinical catalysts:

• iPSC tools: Axol ($2.8M) to expand US ops and manufacturing scale.
• RNA/gene expression: Circio (NOK 65M) oversubscribed rights issue to accelerate circVec platform.
• AI platforms and bioprocess: Several larger rounds (Nuclera, Proxima, Vetter programme investments) highlighted in press rundowns point to investor preference for revenue near, de risked picks and shovel plays.

11. Cost, resilience, and the operations agenda

Argon & Co’s survey of 800+ C suite leaders crystallised the macro constraint: rising operating costs are the #1 threat to growth in life sciences; 40% cite raw material price pressure; 49% plan AI investments to drive efficiencies; and supplier diversification is a top hedge. These pressures explain January’s drumbeat of packaging, CDMO, and analytics announcements – not as press release filler, but as strategic levers on COGS, cycle time, and risk.